Progressive and irreversible vision loss is inevitable for the estimated 1 in 3000 Australians born with inherited retinal disease (IRD).
There is currently no treatment available, but an innovative research project at CERA could change that.
Dr Thomas Edwards, Principal Investigator in Retinal Gene Therapy Research
Principal Investigator in Retinal Gene Therapy Research Dr Thomas Edwards and his team are investigating how to treat a particular IRD by replacing the abnormal gene that causes the disorder.
Dr Edwards was involved in a world-first gene therapy trial at Oxford University and he is now bringing that experience to CERA.
"We're looking at one particular gene and developing a strategy to introduce the correct copy of that gene into retinal cells," he says.
"This is gene therapy in its simplest form - rather than trying to fix the gene we're replacing it.
"We've had some successes delivering our gene of interest into cells grown in the lab. It's giving us plenty of encouragement.
"For people with this particular gene affected, the overall goal is a treatment that halts the further degeneration of the retina and consequent loss of vision, or even partially reverses the damage.
"We hope to establish Melbourne as a centre for ocular gene therapy research, and to attract gene therapy clinical trials that target other IRDs or indeed more common diseases with a genetic basis, such as age- related macular degeneration or glaucoma.
"This would give Australians access to cutting-edge treatments not currently available in our country."
This story is reproduced from CERA's 2018 Annual Review.
